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Enhancing Care Through Research

“Effective treatment of children with DIPG is a challenge — we have failed despite 45 years of clinical research. If we find effective ways to treat these children, the findings could apply to other difficult cancers and have a large impact on the field.” - Dr. Javad Nazarian, Children’s Research Institute

Diffuse Intrinsic Pontine Glioma, often just called DIPG, is a neurologically devastating, and in most children, lethal pediatric cancer that develops in the structural tissue of the brainstem, without truly effective therapy.

There are more than 300 cases in the U.S. each year, but its relative rarity actually makes fighting DIPG more challenging. There is little federal research funding for the study of “rare pediatric” diseases, and many oncologists don’t have specialized experience with DIPG or its treatments.

Our DIPG Program: Why Children’s National

Children’s National is one of just a few hospitals with extensive experience in treating DIPG patients, as well as an active research program innovating new, more promising therapies for DIPG.

A multidisciplinary team of specialists and researchers who are dedicated to finding a cure for DIPG.

What is DIPG?

Understanding what Diffuse Intrinsic Pontine Glioma (DIPG) means will help you understand just why this cancer is so hard to beat.

Diffuse – Threaded through healthy brain tissue, a DIPG tumor is not a solid mass. It can’t simply be removed with surgery.

Intrinsic – It’s due to causes within the brain itself. Some studies suggest DIPG tumors start in childhood because kids’ brains are developing so much, so quickly. There’s no research linking DIPG to genetic or environmental causes.

Pontine – The tumor grows in the pons, the part of the brainstem that puts breathing, swallowing, and other basic bodily functions on autopilot. It can also, less frequently, arise in other parts of the brainstem or extend into other areas. The location makes the tumors all the more debilitating and treatment all the more risky.

Glioma – The tumor starts in glial cells, the white matter “glue” that supports and insulates neurons throughout the brain.

Even as outcomes for children with other types of cancer have vastly improved, long-term survival rates for DIPG have been stalled for more than 50 years. Most DIPG patients die within a year of their diagnosis. Less than 10 percent see two birthdays beyond their diagnosis. In the interim, DIPG patients suffer debilitating side effects as the growing tumor presses on the brainstem and makes seeing, walking, and even breathing increasingly difficult.Yet, precisely because it’s one of the most challenging and lethal cancers, the Children’s team is committed to finding new, more effective treatments for DIPG. We also believe that if we conquer DIPG, we’ll unlock cures for many other types of cancer in the process.

DIPG Diagnosis

DIPG Symptoms

Though children of all ages and even a few adults have been diagnosed with DIPG, most new cases are in children four to ten years old. Some research suggests this is because of rapid brain development in those early elementary years. Symptoms first appear when the tumor puts pressure on nerves in the brainstem that control facial, eye, and muscle movement. Parents might first notice that their child is suddenly clumsy, has developed a “lazy eye," or has facial weakness. As the DIPG tumor grows, most patients suffer some combination of:

Problems seeing, such as double vision, drooping eyes, or a “lazy eye”

Drooping on one side of the face

Trouble with movement, particularly on one side

Difficulty talking, chewing, or swallowing

Hearing problems, including sudden deafness

Headaches

Nausea and vomiting caused by brain swelling

Making a DIPG Diagnosis

As with many neurological symptoms, an MRI is the best source to create a detailed image of the brain. If the scan shows a tumor in the pons, its infiltration of healthy tissue and undefined shape will suggest it’s a DIPG brain tumor. Until just a few years ago, doctors relied solely on the child’s symptoms and an MRI to diagnose DIPG. The risk of damaging the brainstem was just too high to do a biopsy of the tumor.However, as our technology gets more and more precise, researchers have determined we can safely biopsy DIPG brain tumors. This important advance also allows us to see the child’s DIPG brain tumor at the genetic level. During the biopsy, a surgeon makes a small hole in the skull and uses a needle to extract a tiny piece of the tumor. The tissue sample can then be tested to determine the type of tumor and its genetic make-up, including mutations. As our research progresses, we hope to be able to target treatments specifically to each tumor’s genomic profile.

DIPG Treatment at Children's National

Surgical removal, also called resection, is not currently an option for these infiltrative tumors. And we have yet to find any chemotherapy drugs effective against DIPG. However, radiation therapy can often kill some of the cancer cells, relieving pressure on the brainstem and some of the child’s symptoms as a result. About 75-85 percent of DIPG patients respond to radiation, improving their quality of life and beating back the cancer in the short term. Children’s National is one of the few hospitals in the nation that conducts brainstem biopsies for DIPG that can guide in identifying a course of treatment personalized to each childGenomics analyses are run to better understand the biology of that specific tumor. Within three weeks, our team of specialists reviews the results and patients with DIPG are placed on therapy that best meets their individual need.

Access to Clinical Trials

As a leader in DIPG research, our patients have access to open clinical trials sponsored by Children’s National, the Pediatric Brain Tumor Consortium, the Pacific Pediatric Neuro-Oncology Consortium and other consortia. We‘re currently part of a nationwide study of new treatments targeting the unique genomic profile of a child’s tumor. The results of this work should significantly improve biopsy-driven treatments for children with DIPG.We’re also actively testing alternate immuno therapeutic approaches in Children’s National patients. We currently lead a national trial investigating checkpoint inhibitors in children with recurrent DIPG.

DIPG Research at Children's National

The DIPG treatments available today can only stunt the tumor’s growth for a few months and temporarily relieve some symptoms. That’s just not good enough.The Children’s National Brain Tumor Institute and Children’s Research Institute are currently mapping out a four-part, three-year research effort to:

Study promising new drugs and therapies

Make treatment more effective and less damaging

Detect tumors sooner

Genetic Targeting

In the first phase of the project, we’ll categorize different types of DIPG tissue at the molecular and genetic level. We’ll then test promising drugs to testing promising drugs and combinations of drugs against each type of tumor.

The drugs that show the best results in mice – increasing survival and stunting tumor growth – will move on to clinical trials in patients.

Novel Drug Approaches

With robust preclinical data, we plan to launch clinical trials of promising new drugs, including drugs developed at Children's.One such drug is designed to change the way genes are expressed or keep specific genes from copying. Until recently they’ve been used only in labs to study genes, but they hold promise in medicine, too.In fact, our DIPG team has developed drugs that specifically target histone mutations in DIPG. These breakthrough molecules have already been shown to kill DIPG cells in the lab. We believe they could do the same in patients, but with fewer side effects.

Immunotherapy

Immunotherapy – treatments that activate the body’s own immune system to attack cancer cells – has been showing promising results against other forms of cancer and could prove effective against DIPG, too. At Children’s, we’re actively testing alternate immunomodulatory therapies in our DIPG patients. We currently lead a national trial investigating checkpoint inhibitors in children with recurrent DIPG. In the future, our researchers aim to identify specific immunologic targets on DIPG cells. By 2019, we hope to start testing T-cell treatments developed in our Center for Cancer and Immunology Research on DIPG brain tumors.

Support Our DIPG Research

With little federal research money for “rare pediatric” diseases, our DIPG research depends on donations. Please help use raise the more than $2 million we need to launch our visionary DIPG research plan.